{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T03:33:57Z","timestamp":1789011237336,"version":"build-2803163510"},"reference-count":26,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2025,1,14]],"date-time":"2025-01-14T00:00:00Z","timestamp":1736812800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["acrjournals.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Arthritis &amp; Rheumatology"],"published-print":{"date-parts":[[2025,5]]},"abstract":"<jats:sec><jats:title>Objective<\/jats:title><jats:p>Two randomized trials for patients with diffuse systemic sclerosis (SSc) demonstrated an overall survival (OS) and event\u2010free survival (EFS) advantage of autologous hematopoietic stem cell transplantation (AHSCT) using CD34+ selected peripheral blood stem cells (PBSCs) compared with monthly cyclophosphamide (CY). We asked if an unmodified PBSC graft followed by maintenance mycophenolate mofetil (MMF) after AHSCT, instead of a CD34+ selected graft, could provide comparable AHSCT outcomes.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Twenty patients with high\u2010risk SSc were enrolled in a prospective, single\u2010arm trial with CY 200 mg\/kg and horse antithymocyte globulin (ATG; CY200\/ATG), followed by unmanipulated autologous PBSC, and then MMF maintenance starting at 2 months after AHSCT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Point estimates of OS and EFS at 5 years after AHSCT were 85% (95% confidence interval [CI] 60.4%\u201394.9%) and 75% (95% CI 50%\u201388.7%), respectively. Median follow\u2010up was 7.5 years (range 5.6\u201311.6) after transplant for living patients. Eight patients (40%) required intensive care unit treatment early after transplant. Early transplant\u2010related mortality occurred in two patients (10%). Five patients developed relapse\/progression of SSc after AHSCT. Four of nine patients with anti\u2010RNA polymerase III antibodies had prior scleroderma renal crisis and the lowest quartile of estimated glomerular filtration rate (eGFR) on study entry; all four patients developed prolonged organ failure\/death early after transplant.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>We observed favorable OS and EFS after AHSCT for patients with SSc, using CY200\/ATG, unmanipulated PBSCs, and MMF posttransplant maintenance, which was comparable to trials with CD34+ graft selection. We identified a possible risk factor, pretransplant low eGFR, for adverse outcomes after AHSCT.<\/jats:p><\/jats:sec>","DOI":"10.1002\/art.43072","type":"journal-article","created":{"date-parts":[[2024,12,3]],"date-time":"2024-12-03T03:44:28Z","timestamp":1733197468000},"page":"571-581","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation for Diffuse Cutaneous Systemic Sclerosis: Identifying Disease Risk Factors for Toxicity and Long\u2010Term Outcomes in a Prospective, Single\u2010Arm Trial"],"prefix":"10.1002","volume":"77","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0625-0858","authenticated-orcid":false,"given":"George E.","family":"Georges","sequence":"first","affiliation":[{"name":"Northwestern University Feinberg School of Medicine, Chicago, Fred Hutchinson Cancer Center and University of Washington  Seattle"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1412-4453","authenticated-orcid":false,"given":"Dinesh","family":"Khanna","sequence":"additional","affiliation":[{"name":"University of Michigan  Ann Arbor"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark H.","family":"Wener","sequence":"additional","affiliation":[{"name":"University of Washington  Seattle"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew G.","family":"Mei","sequence":"additional","affiliation":[{"name":"City of Hope National Medical Center  Duarte California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5070-2535","authenticated-orcid":false,"given":"Maureen D.","family":"Mayes","sequence":"additional","affiliation":[{"name":"University of Texas Houston  Houston"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert W.","family":"Simms","sequence":"additional","affiliation":[{"name":"(current address: Dartmouth Geisel School of Medicine, Hannover, New Hampshire), Boston Medical Center and Boston University  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vaishali","family":"Sanchorawala","sequence":"additional","affiliation":[{"name":"(current address: Dartmouth Geisel School of Medicine, Hannover, New Hampshire), Boston Medical Center and Boston University  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chitra","family":"Hosing","sequence":"additional","affiliation":[{"name":"M. 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Anderson Cancer Center  Houston Texas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzanne","family":"Kafaja","sequence":"additional","affiliation":[{"name":"University of California Los Angeles  Los Angeles"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Attaphol","family":"Pawarode","sequence":"additional","affiliation":[{"name":"University of Michigan  Ann Arbor"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leona A.","family":"Holmberg","sequence":"additional","affiliation":[{"name":"Fred Hutchinson Cancer Center and University of Washington  Seattle"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7043-9161","authenticated-orcid":false,"given":"Jason","family":"Kolfenbach","sequence":"additional","affiliation":[{"name":"University of Colorado Anschutz  Aurora"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7857-2373","authenticated-orcid":false,"given":"Daniel E.","family":"Furst","sequence":"additional","affiliation":[{"name":"University of California Los Angeles  Los Angeles"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1379-9216","authenticated-orcid":false,"given":"Keith M.","family":"Sullivan","sequence":"additional","affiliation":[{"name":"Duke University  Durham North Carolina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suiyuan","family":"Huang","sequence":"additional","affiliation":[{"name":"University of Michigan  Ann Arbor"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ted","family":"Gooley","sequence":"additional","affiliation":[{"name":"Fred Hutchinson Cancer Center  Seattle Washington"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard A.","family":"Nash","sequence":"additional","affiliation":[{"name":"Colorado Blood Cancer Institute  Denver Colorado"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,1,14]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.11073"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/ker269"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.38390"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.200702-326OC"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)00232-4"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S2213-2600(16)30152-7"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2014.6368"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703327"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1136\/annrheumdis-2013-204424"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2102953"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2015.08.030"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2017.56"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrrheum.2009.238"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.38390"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/1346-8138.12722"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.3899\/jrheum.160867"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00296-021-04855-x"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/jcph.2144"},{"key":"e_1_2_7_20_1","doi-asserted-by":"publisher","DOI":"10.1093\/qjmed\/hcm052"},{"key":"e_1_2_7_21_1","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/ker271"},{"key":"e_1_2_7_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/art.42850"},{"key":"e_1_2_7_23_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.230128"},{"key":"e_1_2_7_24_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13075-019-1823-0"},{"key":"e_1_2_7_25_1","doi-asserted-by":"publisher","DOI":"10.1200\/JCO.2006.09.8327"},{"key":"e_1_2_7_26_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V95.11.3273"},{"key":"e_1_2_7_27_1","doi-asserted-by":"publisher","DOI":"10.4161\/cbt.26342"}],"container-title":["Arthritis &amp; 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